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The Food and Drug Administration’s rejection of a gene therapy for hemophilia A on Wednesday surprised many hematology researchers and Wall Street watchers who expected speedy approval for the one-time treatment to end the inherited bleeding disorder.
For one family in Indianapolis active in the hemophilia patient community, the decision was disappointing, but also appreciated.
“It’s a sad day because for a lot of people, they were ready to go,” Michelle Rice, whose two sons have severe forms of hemophilia A, told STAT. But “it’s also a good day,” said Rice, who has a mild case of the disease and serves as chief external affairs officer for the National Hemophilia Foundation, “because I think this community has fought long and hard for safety to be a priority.”
Hemophilia A is a genetic disorder in which the body fails to produce a protein called Factor VIII that is crucial for blood clotting. It affects about 20,000 people in the U.S., almost all men. The bleeding episodes and joint damage it causes are kept under control by frequent infusions costing about $300,000 per...
